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Nulibry fosdenopterin hydrobromide 9.5 mg Injection, Powder, Lyophilized, For Solution, 1 vial — NDC 42358-0295-01 package photo
Label image from the product's FDA listing (DailyMed) — may show a different pack size or an older label revision.

Nulibry fosdenopterin hydrobromide 9.5 mg Injection, Powder, Lyophilized, For Solution, 1 vial — NDC 42358-295-01 (Billing 42358-0295-01)

by Sentynl Therapeutics, Inc. · 1 VIAL, SINGLE-DOSE in 1 CARTON / 1 INJECTION, POWDER, LYOPHILIZED, FOR SOLUTION in 1 VIAL, SINGLE-DOSE

This is a package of 1 vial of Nulibry fosdenopterin hydrobromide 9.5 mg Injection, Powder, Lyophilized, For Solution from Sentynl Therapeutics, Inc., marketed since Mar 2021 and currently FDA-listed. It is this product's only package size.

NDC 42358-0295-01
🏷️ FDA NDC (as labeled) 42358-295-01 billing pads the product segment with a zero
Rx only Brand On market Non-controlled ⇄ Compare with another NDC
🗂️ FDA directory synced Oct 1, 2026 · this listing last changed Jul 24, 2026 · sources: openFDA · FDA label (DailyMed) · FDA Orange & Purple Book · First Databank · CMS NADAC, ASP, Medicare & Medicaid · RxNorm
📋 All sources & update times →

This page is curated, coded, and maintained by

Compiled from public FDA, CMS, DailyMed, NADAC, and related drug data sources. Editorial policy

NDC database record

One package, one record: these facts belong to NDC 42358-295-01 alone.

Record
FDA NDC Directory package listing · Human prescription drug
Code segments
42358 labeler · 295 product · 01 package
Package marketed since
Oct 28, 2022
Sample package
No — commercial package
Listing certified through
Dec 31, 2027
Billing quantity
1 EA per package
Barcode (UPC-A, from the NDC)
3 4235829501 2
FDA record last changed
Jul 24, 2026

Identity & classification

Regulatory identifiers FDA, NLM and CMS codes for this package

FDA NDC (as labeled) 42358-295-01
Product NDC 42358-295
11-digit billing NDC 42358029501
NCPDP billing unit EA — each (per item)
RxCUI 2531292, 2531297
UNII X41B5W735T
Application # NDA214018
SPL Set ID 736aeea3-f206-454d-95d0-fd30964e8aab
Established class (EPC) Cyclic Pyranopterin Monophosphate
Chemical class Cyclic Pyranopterin Monophosphate
DEA schedule Non-controlled
Marketing category NDA
Marketing status On market
FDA listing status Listed (active directory)
Marketing start 2021-03-30
Route INTRAVENOUS
Dosage form INJECTION, POWDER, LYOPHILIZED, FOR SOLUTION
Substance FOSDENOPTERIN HYDROBROMIDE

Drug-database identifiers Medi-Span GPI and First Databank GCN / HICL / AHFS classification

GPI-14 30906430202120
GCN Seq No 081982
GCN 49269
HICL code 047158
Ingredient (HICL) Fosdenopterin Hydrobromide
HIC1 code Z
Therapeutic class — broad (HIC1) Body As A Whole
HIC2 code Z1
Therapeutic class — intermediate (HIC2) Positive Effects On Wide-Spread Tissue
HIC3 code Z10
Therapeutic class — specific (HIC3) Metabolic Disease Enzyme Replacement, Mocd
AHFS code 92:92.00.00
AHFS class Other Miscellaneous Therapeutic Agents
FDB label name NULIBRY 9.5 MG VIAL
FDB brand name Nulibry
Legend status F — Federal legend — prescription drug or device
Quick answers
  • GSN (GCN sequence number): 081982
  • GCN: 49269
  • GPI-14 (Medi-Span): 30906430202120
  • HICL (First Databank): 047158
  • AHFS class code: 92:92.00.00
  • RxCUI (RxNorm): 2531292
Why two NDCs? The FDA registers this code as 42358-295-01 — a 5-3-2 layout, and that's what's printed on the package and shown on DailyMed. For insurance claims, every NDC is standardized to a uniform 11-digit 5-4-2 format by adding a zero to the product segment → 42358-0295-01. Same drug, same package — only the format differs.
Where does this data come from?
Identifiers from the FDA openFDA NDC Directory and Structured Product Labeling; RxCUI from RxNorm (NLM); GPI from Medi-Span; GCN / HIC / AHFS / legend from First Databank.

RxNorm drug class

This medicine belongs to the Cyclic Pyranopterin Monophosphate class.

Pharmacologic class Cyclic Pyranopterin Monophosphate
Drug family (ATC) Various alimentary tract and metabolism products
Where does this data come from?
Therapeutic classes from RxNorm RxClass (U.S. National Library of Medicine) — Established Pharmacologic Class (FDA), ATC drug family (WHO) and mechanism of action, matched by this product’s RxCUI.

Clinical

Label name NULIBRY 9.5 MG VIAL Ingredient Fosdenopterin Hydrobromide

Supplement & herbal interactions

🌿 Drug–supplement interaction checker →
Where does this data come from?
Plain-language summary from MedlinePlus (U.S. National Library of Medicine); supplement & herbal interactions and nutrient depletion data from the Natural Medicines database; our full guide is HelloPharmacist editorial content.

Pricing

A drug doesn't have one price. Each row is a different public payment system, and none is what you'd pay at the counter — that depends on your insurance. The ⓘ on each row explains what it measures.

Price systemPer mLPer package
Retail pharmacies payNADAC · weekly Not in the retail survey — common for institutional, discontinued, or low-volume packs.
Medicaid paysCMS SDUD · 12 mo No recent Medicaid claims on file for this NDC — rare and low-volume NDCs are suppressed in the public data.
Medicare drug plans payPart D · quarterly No Part D plan price is available for this NDC in our data.
Medicare Part B allowsASP · J1809 $17.736 / J1809 unit —
Where does this data come from?
NADAC (National Average Drug Acquisition Cost) is the CMS weekly pharmacy-acquisition-cost survey — what pharmacies pay. ASP (Average Sales Price) is the CMS Medicare Part B drug-payment file, published quarterly. Medicaid pays is computed by us from CMS State Drug Utilization Data (total reimbursed ÷ units, trailing 12 months) — gross of rebates and inclusive of dispensing fees, so it reflects what Medicaid paid, not an acquisition cost. Medicare drug plans pay is the median negotiated point-of-sale unit cost across plans listing this NDC in the CMS quarterly Prescription Drug Plan pricing files, before rebates. The VA pays is the federal contract price (FSS, and the statutory Big 4 ceiling where listed) from the VA National Acquisition Center pharmaceutical price file. All are free public government data; each measures a different payer, so the figures are not directly comparable.

Billing & reimbursement

FDA NDC (as labeled)42358-295-01
11-digit billing NDC42358-0295-01
Format5-3-2 as registered → padded to 5-4-2 for billing (zero added to the product segment)
HCPCS J-codeJ1809
DescriptorINJECTION, FOSDENOPTERIN, 0.1 MG
Billing units / pkg95 units
How the units are derivedThis package is 1 EA; the HCPCS unit is 0.1 MG, so one package = 95 billing units.
Crosswalk sourcePDAC NDC-HCPCS crosswalk (DME MAC / DMEPOS)
Where does this data come from?
The HCPCS J-code crosswalk comes from the CMS ASP NDC-HCPCS crosswalk and the DMEPDAC (DME MAC) NDC-HCPCS crosswalk — free public CMS data. Billing units are derived from the code’s descriptor and the package amount.

Packaging — all sizes for this product

Package NDCDescription Marketing startMarketing endStatus
42358-0295-01 You're viewing this Main listing 1 VIAL, SINGLE-DOSE in 1 CARTON / 1 INJECTION, POWDER, LYOPHILIZED, FOR SOLUTION in 1 VIAL, SINGLE-DOSE 2023-04-15 — Active

Therapeutic equivalents

ProductLabelerPackNADAC/unitTEStatusPrice vs. this
Nulibry 9.5 mgthis 42358-0295-01 Sentynl 1 vial — — FDA listed —
About this product: this is the brand-name version. We did not find an FDA-approved generic match for this exact strength, form and route.
Where does this data come from?
Equivalents are other NDCs of the same ingredient, form and route from the openFDA NDC Directory, ranked least-expensive-first by NADAC. Therapeutic-equivalence (AB) ratings come from the FDA Orange Book; biologics use the FDA Purple Book for biosimilar & interchangeable status.

Availability & generic status

🏛️
2021
First FDA approval
Feb 2021
📍
2026
Currently FDA-listed
5 years listed
🛡️
2029
Latest patent/protection listed
not a guaranteed launch date
🔒No FDA-approved generic found

We did not find an FDA-approved generic match for this exact strength, form and route. Patent/protection dates below may affect future generic timing.

🛡️ Latest patent/protection date listed: FDA patent/protection data lists protections through Apr 2029. This may affect when a full generic version becomes widely available, but it is not a guaranteed launch date.
📅 FDA approved Feb 26, 2021 RLD RS ⏳ ~2.5 yr to latest listed protection

Why the date isn’t exact: Generic timing can change because patents may be challenged, settled, licensed, added, removed, or worked around with a narrower label — and FDA approval does not always mean a pharmacy can get the generic today.

Patents & exclusivity — FDA Orange Book
US 7504095 — method of use (U-3092)
Exclusivity NCE
Exclusivity ODE-342
2021 2023 2025 2027 2029
Today
LOE
Substance patent Formulation patent Method-of-use patent Exclusivity Pediatric +6mo
🏛️FDA exclusivity
FDA-granted marketing protection. It’s separate from patents and may be shorter than patent protection.
🧪Product / substance patents
Patents covering the active ingredient, product, formulation, or related drug features.
🎯Method-of-use patents
Patents covering specific approved uses. These can sometimes be carved out with a “skinny label,” but not always.
🛈 What do these terms mean?
Patent
Legal protection listed in the Orange Book that may delay generic approval or launch. Issued by the U.S. Patent & Trademark Office.
Substance patent
Covers the active drug molecule itself — the hardest to design around. A generic generally can’t launch until it expires.
Formulation (product) patent
Covers a specific formulation or dosage form. A generic can sometimes work around it with a different formulation.
Method-of-use patent
A patent covering one specific approved use of the drug — not necessarily the whole molecule. A generic can sometimes launch with a “skinny label” that carves out the protected use and keeps the others.
Skinny label
A generic label that omits a still-patented use when the FDA allows it — letting a generic reach the market for the unprotected uses.
Exclusivity
FDA-granted marketing protection, separate from patents — e.g. 5-yr new chemical entity, 7-yr orphan drug, or a +6-month pediatric extension.
Paragraph IV
A generic applicant’s formal challenge to a listed patent. It can potentially lead to earlier generic entry, but often involves litigation or a settlement.
RLD / RS
Reference Listed Drug — the brand product the FDA uses as the reference for generic applications. Reference Standard — the product the FDA expects generics to compare against in bioequivalence testing.
TE / AB rating
FDA therapeutic-equivalence rating. An AB rating generally means the FDA considers a generic therapeutically equivalent to — and substitutable for — the brand.
LOE (loss of exclusivity)
The latest patent or exclusivity currently listed — the loss-of-exclusivity / latest-listed-protection date shown on this page. Paragraph-IV challenges and settlements can move the real date earlier; FDA approval and a manufacturer’s decision to market can move it later.

Built from the FDA Orange Book. The bars above are scaled to each protection’s expiry; the red LOE marker is the last one to lapse.

Listed patents (1)
PatentTypeUse codeExpires
US 7504095 ↗ Method of use U-3092 Apr 9, 2029
FDA exclusivity
CodeWhat it grantsExpires
NCENew Chemical Entity (5-year)Feb 26, 2026
ODE-342Orphan Drug Exclusivity (7-year)Feb 26, 2028
Common questions
Is there a generic version of NULIBRY 9.5 MG VIAL?
No FDA-approved generic equivalent is currently listed in the FDA Orange Book for NULIBRY 9.5 MG VIAL. Based on the patents and exclusivity currently listed, the Orange Book estimate is that full-label generic entry may be delayed until Apr 2029 — an estimate, not a guaranteed launch date.
The FDA approved a generic — why can’t I get it at my pharmacy yet?
FDA approval and pharmacy availability are two different things. The FDA can approve a generic years before it actually reaches pharmacies, because the brand company may still hold patents or have a settlement that delays the launch. A manufacturer also has to choose to make and sell it, and have supply ready. So a drug can be “FDA-approved generic exists” and still be brand-only at the counter today.
Why do different websites show different generic release dates?
Generic availability is not based on one single date. Some sources use the first exclusivity expiration, some use the last product patent, and others use the latest method-of-use patent. Patent challenges, settlements, licenses, and label carve-outs can also change the real-world launch date. This page shows the underlying Orange Book dates so you can see why estimates may differ.
What does “FDA listed” mean?
It means the product appears in the FDA’s official NDC directory. That’s a good sign a product exists and is intended for the U.S. market, but on its own it does not confirm a pharmacy can fill it today. Where we have recent retail pricing data (NADAC) for a product, we label it “Availability likely” instead.
What does a patent or protection date mean here?
It’s the latest date currently listed in the FDA Orange Book for a patent or exclusivity on the brand product. It can affect when a full generic version becomes widely available — but it is not a guaranteed generic launch date. Generics sometimes arrive earlier (through a settlement or patent challenge) or later (a manufacturer still has to make and sell one).
What does “current Orange Book estimate” mean?
It means we are using the latest patent and exclusivity dates currently listed in the FDA Orange Book. It is not a guaranteed launch date.
Can a generic come out before the last patent expires?
Sometimes. A generic company may challenge a patent, settle with the brand manufacturer, receive a license, or obtain approval with a narrower label that avoids a patented use. In other cases, the last listed protection may delay full-label generic competition.
Can a generic come out after the listed dates?
Yes. Even after patents or exclusivity expire, a generic still needs FDA approval and a manufacturer must choose to market it. Supply, litigation, business decisions, or regulatory issues can delay actual availability.
What is the difference between patents and exclusivity?
Patents are legal protections usually issued by the U.S. Patent and Trademark Office. FDA exclusivity is marketing protection granted by the FDA. They are separate, and either one can affect generic timing.
Why are there multiple patent dates?
One drug can have several patents covering different things: the active ingredient, a formulation, a manufacturing process, or a specific approved use. That is why a page may show several expiration dates instead of one simple generic date.
Built from FDA Orange Book patent and exclusivity data. Dates are refreshed from public FDA data when available; the marker is max(latest patent expiry, latest exclusivity expiry). Paragraph-IV settlements and first-filer 180-day exclusivity can shift the real date; a method-of-use patent may allow an earlier skinny-label generic for non-protected indications. Generic launch timing is an estimate, not a guarantee.
Where does this data come from?
Patents and exclusivity from the FDA Orange Book (small-molecule drugs), refreshed from public FDA data. Generic launch timing is an estimate, not a guarantee.

Inactive Ingredients / Excipients

Inactive ingredients, also called excipients, are components of the drug product other than the active ingredient. They may include fillers, dyes, coatings, preservatives, flavors, or other formulation ingredients.

💡 Tap an ingredient (hover on desktop) to see what it is and why it’s used.

  • 10 mg UNII PQ6CK8PD0R
    Ascorbic acid, also known as vitamin C, is a white crystalline powder. It serves as an antioxidant to prevent degradation of other ingredients and may also function as a preservative in the formulation.
  • UNII QTT17582CB
    A strong acid used to adjust and maintain the proper pH level in liquid medicines, ensuring stability and preventing breakdown of active ingredients.
  • 187.5 mg UNII 3OWL53L36A
    A natural sugar alcohol derived from seaweed or synthesized in the lab. It's used as a filler to add bulk, a sweetener in sugar-free formulas, and a disintegrant to help tablets break apart in the stomach.
  • UNII 55X04QC32I
    A strong alkaline chemical used to adjust and maintain the pH balance of liquid medicines. It helps keep the medicine stable and ensures it stays effective during storage.
  • 62.5 mg UNII C151H8M554
    A natural sugar derived from sugar cane or sugar beets. It's used as a sweetener, filler, and binder to improve taste, add bulk, and help hold tablet or capsule ingredients together.

5 inactive ingredients listed in the exact product block matched to this NDC.

Where does this data come from?
Data sourced from official FDA Structured Product Labeling (SPL) via DailyMed — ingredient classCode="IACT" elements from the exact product block matched by this NDC. Label-section narrative from DailyMed / the openFDA label index is shown separately when available.

Inactive ingredient FAQ

Are inactive ingredients the same for every manufacturer?
No. Inactive ingredients can differ by manufacturer, dosage form, strength, and package / product version.
Why might an inactive ingredient be missing?
Some SPLs do not provide a complete structured inactive-ingredient list, and older or unusual labels may only include the information in narrative text.
Can inactive ingredients matter?
Yes. They can matter for allergies, intolerances, dyes, gluten / lactose concerns, preservatives, and formulation differences — but confirm with a pharmacist or the manufacturer when it’s clinically important.

Manufacturer & labeler

LabelerSentynl Therapeutics, Inc.
Application holderSENTYNL THERAPEUTICS INC
FDA applicationNDA214018 (NDA)
Labeler code42358
First marketedMar 2021
Product typeHuman Prescription Drug
Portfolio4 products on file

More NDCs from Sentynl Therapeutics, Inc. labeler code 42358

The labeler markets the product; the application holder owns the FDA approval. They’re often the same company but can differ (e.g. a repackager or an authorized generic). A mailing address / phone appears here when the manufacturer includes it in the product’s FDA label (not all do).
Where does this data come from?
Labeler, application holder and registered establishment from the FDA openFDA NDC Directory and Drugs@FDA; address/contact from the product’s FDA label.

Full prescribing information FDA SPL

The complete FDA label for this product — the official prescribing information, verbatim, section by section. Very long sections are excerpted here and marked; the full text is on DailyMed (linked in the sources below). Jump with a chip, search within the label, or expand everything.
🎯 Indications and Usage ⏱️ Dosage and Administration 💊 Dosage Forms and Strengths ⛔ Contraindications ⚠️ Warnings and Cautions 🤒 Adverse Reactions 👥 Use in Specific Populations 🤰 Pregnancy 🧒 Pediatric Use 🧓 Geriatric Use 🧬 Clinical Pharmacology 🧬 Mechanism of Action 📦 How Supplied / Storage and Handling 📦 Storage and Handling 📋 Description 💬 Information for Patients 🧬 Pharmacokinetics 🧬 Pharmacodynamics 🔬 Clinical Studies 🧪 Nonclinical Toxicology 📄 Carcinogenesis, Mutagenesis, Impairment of Fertility 📄 Patient Package Insert 📄 Recent Major Changes 📄 Package Label / Principal Display Panel
🎯 Indications and Usage 47 words ▾

1 INDICATIONS AND USAGE NULIBRY is indicated to reduce the risk of mortality in patients with molybdenum cofactor deficiency (MoCD) Type A. NULIBRY is cyclic pyranopterin monophosphate (cPMP) indicated to reduce the risk of mortality in patients with molybdenum cofactor deficiency (MoCD) Type A. ( 1 )

⏱️ Dosage and Administration ~3 min read ▾

2 DOSAGE AND ADMINISTRATION Start NULIBRY if known or presumed MoCD Type A. Promptly discontinue if MoCD Type A is not confirmed by genetic testing. ( 2.1 ) NULIBRY is intended for administration by a healthcare provider.

If deemed appropriate by a healthcare provider, NULIBRY may be administered at home by the patient's caregiver. ( 2.2 ) See the table below for the recommended dosage in patients less than one year of age. Administer NULIBRY by intravenous infusion once daily.

For dose volumes less than 2 mL, consider administering by slow intravenous push. ( 2.3 ) Titration Duration Preterm Neonates (Gestational Age Less than 37 Weeks) Term Neonates (Gestational Age 37 weeks and Above) Initial Dosage 0.4 mg/kg once daily 0.55 mg/kg once daily Dosage at Month 1 and Month 2 0.7 mg/kg once daily 0.75 mg/kg once daily Dosage at Month 3 to Month 12 0.9 mg/kg once daily 0.9 mg/kg once daily Recommended Dosage in Patients One Year of Age or Older: 0.9 mg/kg given as an intravenous infusion once daily.

( 2.3 )

2.1Patient Selection Start NULIBRY if the patient has a diagnosis or presumptive diagnosis of MoCD Type A. In patients with a presumptive diagnosis of MoCD Type A, confirm the diagnosis of MoCD Type A immediately after initiation of NULIBRY treatment. In such patients, discontinue NULIBRY if the MoCD Type A diagnosis is not confirmed by genetic testing.

2.2Important Recommendation Prior to NULIBRY Treatment NULIBRY is intended for administration by a healthcare provider. If deemed appropriate by a healthcare provider, NULIBRY may be administered at home by the patient's caregiver. If NULIBRY can be administered by a caregiver/patient, advise them to read the detailed instructions on the preparation, administration, storage, and disposal of NULIBRY for caregivers [see Instructions for Use ].

2.3Recommended Dosage and Administration Recommended Dosage and Administration in Patients Less Than One Year of Age (by gestational age) The recommended dosage regimen of NULIBRY in patients less than one year of age (by gestational age), is based on actual body weight as shown in Table 1 . Administer NULIBRY by intravenous infusion once daily. For dose volumes less than 2 mL, consider administering by slow intravenous push using syringe administration [ see Dosage and Administration ( 2.4 ) ] .

Table 1 Recommended Initial Dosage and Titration Schedule of NULIBRY for Patients Less Than One Year of Age by Gestational Age Titration Duration Preterm Neonates (Gestational Age Less than 37 Weeks) Term Neonates (Gestational Age 37 Weeks and Above) Initial Dosage 0.4 mg/kg once daily 0.55 mg/kg once daily Dosage at Month 1 and Month 2 0.7 mg/kg once daily 0.75 mg/kg once daily Dosage at Month 3 to Month 12 0.9 mg/kg once daily 0.9 mg/kg once daily Recommended Dosage and Administration in Patients One Year of Age or Older For patients one year of age or older, the recommended dosage of NULIBRY is 0.9 mg/kg (based on actual body weight) administered as an intravenous infusion once daily.

Missed Dose If a NULIBRY dose is missed, administer the missed dose as soon as possible. Administer the next scheduled dose at least 6 hours after the administration of the missed dose.

2.4Preparation and Administration Instructions Preparation NULIBRY must be reconstituted prior to use. Use aseptic technique during preparation and follow these instructions: Calculate the dose based on the patient's weight to determine the number of vials needed and total reconstituted dose volume. More than one vial may be needed to achieve the calculated dose.

Remove the required number of vials from the freezer to allow them to reach room temperature (by hand warming for 3 to 5 minutes or exposing to ambient air for approximately 30 minutes). Reconstitute each NULIBRY vial with 5 mL of Sterile Water for Injection, USP. Gently swirl the vial continuously until the powder is completely dissolved.

DO NOT shake. After reconstitution, the final concentration of NULIBRY reconstitu… [Excerpted — this section continues on DailyMed.]

💊 Dosage Forms and Strengths 48 words ▾

3 DOSAGE FORMS AND STRENGTHS For injection: 9.5 mg of fosdenopterin, as a white to pale yellow lyophilized powder or cake in a single-dose vial for reconstitution. For injection: 9.5 mg of fosdenopterin as a lyophilized powder or cake in a single-dose vial for reconstitution. ( 3 )

⛔ Contraindications 7 words ▾

4 CONTRAINDICATIONS None. None. ( 4 )

⚠️ Warnings and Cautions 152 words ▾

5 WARNINGS AND PRECAUTIONS Potential for Photosensitivity : Advise patients/caregivers to avoid patient exposure to sunlight, and to have the patient wear sunscreen, protective clothing, and sunglasses when exposed to the sun. If photosensitivity occurs, advise caregivers/patients to seek medical attention immediately and consider a dermatological evaluation. ( 5.1 , 13.2 )

5.1Potential for Photosensitivity Animal studies have identified that NULIBRY has phototoxic potential [ see Nonclinical Toxicology ( 13.2 ) ]. Advise NULIBRY-treated patients or their caregivers to avoid or minimize patient exposure to direct sunlight and artificial UV light exposure (i.e., UVA or UVB phototherapy) and adopt precautionary measures (e.g., have the patient wear protective clothing and hats, use broad spectrum sunscreen with high sun protection factor (SPF) in patients 6 months of age and older, and wear sunglasses when exposed to the sun).

If photosensitivity occurs, advise caregivers/patients to seek medical attention immediately and consider a dermatological evaluation.

🤒 Adverse Reactions ~3 min read ▾

6 ADVERSE REACTIONS The most common adverse reactions (>25%) were catheter-related complications, pyrexia, viral infection, pneumonia, otitis media, vomiting, cough/sneezing, viral upper respiratory infection, gastroenteritis, bacteremia, and diarrhea. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Sentynl Therapeutcis, Inc. at 888-507-5206 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch .

6.1Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. Overview of Safety Evaluation The safety of NULIBRY was assessed in 37 pediatric patients and healthy adults who received at least one intravenous infusion of NULIBRY or an E. coli derived non-salt, anhydrous form of cPMP (recombinant cPMP or rcPMP, which has the same active moiety and therefore the same biologic activity as NULIBRY).

Of these 37 patients/healthy adults, 13 were pediatric patients with MoCD Type A in Studies 1, 2, and 3 [ see Clinical Studies ( 14 )] , 6 were pediatric patients with presumptive MoCD Type A but who were later confirmed to not have MoCD Type A, and 18 were healthy adults (without MoCD Type A) in a Phase 1 study. Adverse Reactions Assessment of adverse reactions for NULIBRY is based on data from two open-label, single-arm studies, Study 1 (n=8) and Study 2 (n=1), in patients with a confirmed diagnosis of MoCD Type A (8 of the 9 patients were previously treated with rcPMP).

In these studies, patients received a daily intravenous infusion of NULIBRY. The median exposure to NULIBRY was 4.3 years and ranged from 8 days to 5.6 years [ see Clinical Studies ( 14 )]. In these studies, 44% of patients were males and 56% were females, 67% were White and 33% were Asian.

The mean age was 14 days and ranged from 1 day to 69 days at time of first infusion. Table 2 presents the most common adverse reactions that occurred in NULIBRY-treated patients in Studies 1 and 2. Table 2 Common Adverse Reactions Reported in Two or More NULIBRY-Treated Patients with MoCD Type A (Studies 1 and 2) Adverse Reactions NULIBRY-Treated Patients (N=9) n (%) Abbreviations: MoCD = molybdenum cofactor deficiency 1 Catheter-related complications included complication associated with device, catheter site abscess, catheter site discharge, catheter site extravasation, catheter site pain, catheter site infection, catheter site inflammation, device dislocation, device leakage, device occlusion, and vascular device infection.

Catheter-related complications 1 8 (89%) Pyrexia 7 (78%) Viral infection 5 (56%) Pneumonia 4 (44%) Otitis Media 4 (44%) Vomiting 4 (44%) Cough/Sneezing 4 (44%) Upper viral respiratory infection 3 (33%) Gastroenteritis 3 (33%) Diarrhea 3 (33%) Bacteremia 3 (33%) Abdominal pain 2 (22%) Influenza 2 (22%) Lower respiratory tract infection 2 (22%) Viral tonsillitis 2 (22%) Oropharyngeal pain 2 (22%) Rash maculo-papular 2 (22%) Anemia 2 (22%) Eye swelling 2 (22%) Seizure 2 (22%) Agitation 2 (22%) Safety data are also available from 10 patients with MoCD Type A who received rcPMP in Study 3 (an observational study) [ see Clinical Studies ( 14 )].

The median time on rcPMP treatment was 1.5 years and ranged from 6 days to 4.4 years. In Study 3, the patient population was evenly distributed between males and females with a mean age of 18 days (range 1, 69) at time of first infusion, 70% were White, and 30% were Asian. In Study 3, one patient died of necrotizing enterocolitis.

Adverse reactions for the rcPMP-treated patients were similar to the NULIBRY-treated patients, except for the following additional adverse reactions that were reported in more than one patient: sepsis, oral candidiasis, varicella, fungal skin infection, and eczema.

👥 Use in Specific Populations ~2 min read ▾

8 USE IN SPECIFIC POPULATIONS

8.1Pregnancy Risk Summary There are no available data on NULIBRY use in pregnant females to evaluate for a drug-associated risk of major birth defects, miscarriage, or other adverse maternal or fetal outcomes. In animal reproduction studies, no adverse developmental outcomes occurred at dose exposures 32-89 times the human exposure at the maximum recommended human dose (MRHD) of 0.9 mg/kg/day (see Data ). The background risk of major birth defects and miscarriage for the indicated population is unknown.

All pregnancies have a background risk of birth defect, loss, or other adverse outcomes. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively. Data Animal Data In an embryofetal development study, intravenous administration of fosdenopterin to pregnant rabbits once daily during the period of organogenesis (GD 7 to GD 20) at doses up to 200 mg/kg/day (approximately 89 times the human exposure at the MRHD, based on AUC) did not result in adverse developmental effects.

In a pre- and postnatal development study, subcutaneous administration of fosdenopterin to pregnant mice once daily throughout pregnancy and lactation (GD 6 to Lactation Day 20) at doses of 50, 100, or 200 mg/kg/day (up to 32 times the human exposure at the MRHD, based on AUC) did not result in adverse developmental effects.

8.2Lactation Risk Summary There are no human or animal data available to assess the presence of NULIBRY or its metabolites in human milk, the effects on the breastfed infant, or the effects on milk production for the mother. The developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for NULIBRY and any potential adverse effects on the breastfed infant from NULIBRY or from the underlying maternal condition.

8.4Pediatric Use Safety and effectiveness of NULIBRY for the treatment of MoCD Type A have been established in pediatric patients starting from birth. Use of NULIBRY for this indication is supported by evidence from two open-label studies (Studies 1 and 2) and one observational study (Study 3), in which 13 pediatric patients aged birth to 6 years of age were treated with NULIBRY or rcPMP. Pediatric use information is discussed throughout the labeling.

Animal studies have identified that NULIBRY has phototoxic potential. Advise NULIBRY-treated patients or their caregivers to avoid patient exposure to direct sunlight and artificial UV light exposure (i.e., UVA or UVB phototherapy) and adopt precautionary measures [ see Warnings and Precautions ( 5.1 ) and Nonclinical Toxicology ( 13.2 ) ].

8.5Geriatric Use MoCD Type A is largely a disease of pediatric patients. Clinical studies of NULIBRY did not include patients 65 years of age and older.

8.6Adult Use The safety and effectiveness of NULIBRY for the treatment of adults with MoCD Type A have been established. Use of NULIBRY in adults for this indication is based on an adequate and well- controlled clinical investigation in pediatric patients [ see Clinical Studies ( 14 ) ].

🤰 Pregnancy ~1 min read ▾

8.1Pregnancy Risk Summary There are no available data on NULIBRY use in pregnant females to evaluate for a drug-associated risk of major birth defects, miscarriage, or other adverse maternal or fetal outcomes. In animal reproduction studies, no adverse developmental outcomes occurred at dose exposures 32-89 times the human exposure at the maximum recommended human dose (MRHD) of 0.9 mg/kg/day (see Data ). The background risk of major birth defects and miscarriage for the indicated population is unknown.

All pregnancies have a background risk of birth defect, loss, or other adverse outcomes. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively. Data Animal Data In an embryofetal development study, intravenous administration of fosdenopterin to pregnant rabbits once daily during the period of organogenesis (GD 7 to GD 20) at doses up to 200 mg/kg/day (approximately 89 times the human exposure at the MRHD, based on AUC) did not result in adverse developmental effects.

In a pre- and postnatal development study, subcutaneous administration of fosdenopterin to pregnant mice once daily throughout pregnancy and lactation (GD 6 to Lactation Day 20) at doses of 50, 100, or 200 mg/kg/day (up to 32 times the human exposure at the MRHD, based on AUC) did not result in adverse developmental effects.

🧒 Pediatric Use 125 words ▾

8.4Pediatric Use Safety and effectiveness of NULIBRY for the treatment of MoCD Type A have been established in pediatric patients starting from birth. Use of NULIBRY for this indication is supported by evidence from two open-label studies (Studies 1 and 2) and one observational study (Study 3), in which 13 pediatric patients aged birth to 6 years of age were treated with NULIBRY or rcPMP. Pediatric use information is discussed throughout the labeling.

Animal studies have identified that NULIBRY has phototoxic potential. Advise NULIBRY-treated patients or their caregivers to avoid patient exposure to direct sunlight and artificial UV light exposure (i.e., UVA or UVB phototherapy) and adopt precautionary measures [ see Warnings and Precautions ( 5.1 ) and Nonclinical Toxicology ( 13.2 ) ].

🧓 Geriatric Use 27 words ▾

8.5Geriatric Use MoCD Type A is largely a disease of pediatric patients. Clinical studies of NULIBRY did not include patients 65 years of age and older.

🧬 Clinical Pharmacology ~2 min read ▾

12 CLINICAL PHARMACOLOGY

12.1Mechanism of Action Patients with MoCD Type A have mutations in the MOCS1 gene leading to deficient MOCS1A/B dependent synthesis of the intermediate substrate, cPMP. Substrate replacement therapy with NULIBRY provides an exogenous source of cPMP, which is converted to molybdopterin. Molybdopterin is then converted to molybdenum cofactor, which is needed for the activation of molybdenum-dependent enzymes, including sulfite oxidase (SOX), an enzyme that reduces levels of neurotoxic sulfites.

12.2Pharmacodynamics In MoCD Type A, the lack of effective SOX leads to elevated levels of the neurotoxic sulfite, S-sulfocysteine (SSC). Treatment with NULIBRY resulted in a reduction in the level of urinary SSC normalized to creatinine and the reduction was sustained with long-term treatment with NULIBRY [ see Clinical Studies ( 14 ) ] . Cardiac Electrophysiology At the maximum approved recommended dose, NULIBRY did not prolong the QT interval to any clinically relevant extent.

12.3Pharmacokinetics The pharmacokinetics of fosdenopterin in healthy adult subjects following a single intravenous NULIBRY infusion are summarized in Table 3 . The area under the plasma concentration-time curve (AUC) and the maximum plasma concentration (C max ) of fosdenopterin increased in an approximately proportional manner with increasing doses. Table 3 Mean (SD) Pharmacokinetic Parameters Following A Single Intravenous Dose of Fosdenopterin in Healthy Subjects 1 0.075 mg/kg, 0.24 mg/kg, and 0.68 mg/kg doses are 0.08, 0.27, and 0.76 times the recommended maximum dose, respectively.

Parameter 0.075 mg/kg 1 0.24 mg/kg 1 0.68 mg/kg 1 C max (ng/mL) 285 (57) 873 (99) 2800 (567) AUC 0-inf (ng*h/mL) 523 (75) 1790 (213) 5960 (1820) Distribution The volume of distribution (V d ) of fosdenopterin was approximately 300 mL/kg. The plasma protein binding of fosdenopterin ranged from 6 to 12%. Elimination The mean total body clearance (CL) of fosdenopterin ranged from 167 to 195 mL/h/kg.

The mean half-life of fosdenopterin ranged from 1.2 to 1.7 hours. Metabolism Fosdenopterin is predominantly metabolized through nonenzymatic degradation processes to Compound Z, an inactive oxidation product of endogenous cPMP. Excretion Renal clearance of fosdenopterin accounts for approximately 40% of total body clearance.

Specific Populations The effect of renal and hepatic impairment on the pharmacokinetics of fosdenopterin is unknown. Pediatric Patients Pharmacokinetic properties of fosdenopterin in pediatric MoCD Type A patients are similar to healthy adult subjects. Drug Interaction Studies In Vitro Studies Cytochrome P450 (CYP) Enzymes : Fosdenopterin does not inhibit CYP1A2, CYP2B6, CYP2C8, CYP2C9, CYP2C19, CYP2D6, or CYP3A4/5.

Fosdenopterin does not induce CYP1A2, CYP2B6, or CYP3A4. Transporter Systems : Fosdenopterin is a weak inhibitor of MATE2-K and OAT1, but does not inhibit P-gp, BCRP, OATP1B1, OATP1B3, OCT2, OAT3, and MATE1. Fosdenopterin is a weak substrate for MATE1, but is not a substrate of P-gp, BCRP, OATP1B1, OATP1B3, OCT2, OAT1, OAT3, or MATE2-K.

🧬 Mechanism of Action 70 words ▾

12.1Mechanism of Action Patients with MoCD Type A have mutations in the MOCS1 gene leading to deficient MOCS1A/B dependent synthesis of the intermediate substrate, cPMP. Substrate replacement therapy with NULIBRY provides an exogenous source of cPMP, which is converted to molybdopterin. Molybdopterin is then converted to molybdenum cofactor, which is needed for the activation of molybdenum-dependent enzymes, including sulfite oxidase (SOX), an enzyme that reduces levels of neurotoxic sulfites.

📦 How Supplied / Storage and Handling 91 words ▾

16 HOW SUPPLIED/STORAGE AND HANDLING How Supplied NULIBRY (fosdenopterin) for injection is a white to pale yellow lyophilized powder or cake in a single-dose clear glass vial for reconstitution. Each NULIBRY vial contains 9.5 mg of fosdenopterin. Each carton of NULIBRY contains one vial (NDC 42358-295-01).

Components are not made with natural rubber latex. Storage Store NULIBRY frozen between -25°C and -10°C (-13°F and 14°F). Store vial in original carton to protect from light.

For storage recommendations for the reconstituted solution [ see Dosage and Administration ( 2.6 ) ] .

📦 Storage and Handling 37 words ▾

Storage Store NULIBRY frozen between -25°C and -10°C (-13°F and 14°F). Store vial in original carton to protect from light. For storage recommendations for the reconstituted solution [ see Dosage and Administration ( 2.6 ) ] .

📋 Description 156 words ▾

11 DESCRIPTION NULIBRY (fosdenopterin) for injection is cyclic pyranopterin monophosphate (cPMP). Fosdenopterin is present as a dihydrate of the hydrobromide salt with the chemical name (4a R ,5a R ,11a R ,12a S )-8-amino-4a,5a,6,9,11,11a,12,12a-octahydro-2,12,12-trihydroxy-1,3,2-dioxaphosphorino[4',5':5,6]pyrano[3,2- g ]pteridin-10(4 H )-one 2-oxide. Fosdenopterin hydrobromide as a dihydrate is a crystalline solid.

The molecular formula is C 10 H 14 N 5 O 8 P • HBr • 2H 2 O and the molecular weight is 480.16. The chemical structure is: NULIBRY is supplied as a sterile, preservative-free, white to pale yellow lyophilized powder or cake in a single-dose, clear glass vial for reconstitution for intravenous infusion. Each vial contains 9.5 mg fosdenopterin (equivalent to 12.5 mg fosdenopterin hydrobromide as a dihydrate).

Each vial also contains the following inactive ingredients: 10 mg ascorbic acid USP, 187.5 mg mannitol USP, and 62.5 mg sucrose NF. Sodium hydroxide NF and hydrochloric acid NF are used to adjust pH to 5.0-7.0. chemical structure

💬 Information for Patients 148 words ▾

17 PATIENT COUNSELING INFORMATION Advise patients/caregivers to read the FDA-approved patient labeling (Instructions for Use) and complete the treatment logs as appropriate. Photosensitivity Advise patients and/or caregivers of the potential for photosensitivity reactions and to ensure that the patient avoids or minimizes exposure to sunlight and artificial UV light exposure (i.e., UVA or UVB phototherapy) during use of NULIBRY, uses broad spectrum sunscreen with high sun protection factor (patients 6 months of age and older), and wears clothing, a hat, and sunglasses that protects against sun exposure.

Instruct patients/caregivers to seek medical attention immediately if the patient develops a rash or if they notice symptoms of photosensitivity reactions (redness, burning sensation of the skin, blisters) [ see Warnings and Precautions ( 5.1 ) and Nonclinical Toxicology ( 13.2 ) ] . Manufactured by: Alcami Carolinas Corporation, Charleston, SC 29405 Distributed by: Sentynl Therapeutics, Inc., Solana Beach, CA 92075

🧬 Pharmacokinetics ~1 min read ▾

12.3Pharmacokinetics The pharmacokinetics of fosdenopterin in healthy adult subjects following a single intravenous NULIBRY infusion are summarized in Table 3 . The area under the plasma concentration-time curve (AUC) and the maximum plasma concentration (C max ) of fosdenopterin increased in an approximately proportional manner with increasing doses. Table 3 Mean (SD) Pharmacokinetic Parameters Following A Single Intravenous Dose of Fosdenopterin in Healthy Subjects 1 0.075 mg/kg, 0.24 mg/kg, and 0.68 mg/kg doses are 0.08, 0.27, and 0.76 times the recommended maximum dose, respectively.

Parameter 0.075 mg/kg 1 0.24 mg/kg 1 0.68 mg/kg 1 C max (ng/mL) 285 (57) 873 (99) 2800 (567) AUC 0-inf (ng*h/mL) 523 (75) 1790 (213) 5960 (1820) Distribution The volume of distribution (V d ) of fosdenopterin was approximately 300 mL/kg. The plasma protein binding of fosdenopterin ranged from 6 to 12%. Elimination The mean total body clearance (CL) of fosdenopterin ranged from 167 to 195 mL/h/kg.

The mean half-life of fosdenopterin ranged from 1.2 to 1.7 hours. Metabolism Fosdenopterin is predominantly metabolized through nonenzymatic degradation processes to Compound Z, an inactive oxidation product of endogenous cPMP. Excretion Renal clearance of fosdenopterin accounts for approximately 40% of total body clearance.

Specific Populations The effect of renal and hepatic impairment on the pharmacokinetics of fosdenopterin is unknown. Pediatric Patients Pharmacokinetic properties of fosdenopterin in pediatric MoCD Type A patients are similar to healthy adult subjects. Drug Interaction Studies In Vitro Studies Cytochrome P450 (CYP) Enzymes : Fosdenopterin does not inhibit CYP1A2, CYP2B6, CYP2C8, CYP2C9, CYP2C19, CYP2D6, or CYP3A4/5.

Fosdenopterin does not induce CYP1A2, CYP2B6, or CYP3A4. Transporter Systems : Fosdenopterin is a weak inhibitor of MATE2-K and OAT1, but does not inhibit P-gp, BCRP, OATP1B1, OATP1B3, OCT2, OAT3, and MATE1. Fosdenopterin is a weak substrate for MATE1, but is not a substrate of P-gp, BCRP, OATP1B1, OATP1B3, OCT2, OAT1, OAT3, or MATE2-K.

🧬 Pharmacodynamics 76 words ▾

12.2Pharmacodynamics In MoCD Type A, the lack of effective SOX leads to elevated levels of the neurotoxic sulfite, S-sulfocysteine (SSC). Treatment with NULIBRY resulted in a reduction in the level of urinary SSC normalized to creatinine and the reduction was sustained with long-term treatment with NULIBRY [ see Clinical Studies ( 14 ) ] . Cardiac Electrophysiology At the maximum approved recommended dose, NULIBRY did not prolong the QT interval to any clinically relevant extent.

🔬 Clinical Studies ~3 min read ▾

14 CLINICAL STUDIES The efficacy of NULIBRY for the treatment of patients with MoCD Type A was established based on data from three clinical studies (Studies 1, 2, and 3) that were compared to data from a natural history study. Study 1 Study 1 (NCT02047461) was a prospective, open-label, single-arm, dose escalation study in patients with MoCD Type A who were receiving treatment with rcPMP prior to treatment with NULIBRY. Study 1 included 8 patients, 6 of whom previously participated in Study 3.

The initial NULIBRY dosage was matched to the patient's rcPMP dosage upon entering the study. The NULIBRY dosage was then titrated over a period of 5 months to a maximum dosage of 0.9 mg/kg administered once daily as an intravenous infusion. Study 2 Study 2 (NCT02629393) was a prospective, open-label, single-arm, dose escalation study in one patient with MoCD Type A who had not been previously treated with rcPMP.

The initial dosage of NULIBRY in Study 2 was based on the gestational age of the patient (i.e., 36 weeks). The initial dosage was then incrementally escalated up to a maximum dosage of 0.98 mg/kg administered once daily as an intravenous infusion (1.1 times the maximum approved recommended dosage) [ see Dosage and Administration ( 2.3 ) ]. Study 3 Study 3 was a retrospective, observational study that included 10 patients with a confirmed diagnosis of MoCD Type A who received rcPMP.

Six of these 10 patients were later enrolled in Study 1 to receive treatment with NULIBRY. Efficacy Results The efficacy of NULIBRY and rcPMP were assessed in a combined analysis of the 13 patients with genetically confirmed MoCD Type A from Study 1 (n=8), Study 2 (n=1), and Study 3 (n=4) who received substrate replacement therapy with NULIBRY or rcPMP. Of the 13 treated patients included in the combined analysis, 54% were male, 77% were White and 23% were Asian; the median gestational age was 39 weeks (range 35 to 41 weeks).

Of these 13 treated patients, the age at first dose was ≤ 14 days for 10 patients (with 5 patients initiating treatment at 1 day of age) and ≥ 32 days and < 69 days for the remaining 3 patients. Overall Survival Efficacy was assessed by comparing overall survival in pediatric patients treated with NULIBRY or rcPMP (n=13) with an untreated natural history cohort of pediatric patients with genetically confirmed MoCD Type A who were genotype-matched to the treated patients (n=18). Patients treated with NULIBRY or rcPMP had an improvement in overall survival compared to the untreated, genotype-matched, historical control group ( Table 4 and Figure 1 ).

Results were similar when comparing treated patients with all patients in the untreated natural history cohort with genetically confirmed MoCD Type A (n=37, includes the 18 genotype-matched untreated patients as well as 19 additional untreated patients who were not genotype-matched). Table 4 Overall Survival in Patients with MoCD Type A Treated with NULIBRY or rcPMP Versus Genotype-Matched Untreated Patients in Historical Control Abbreviations: CI=confidence interval; NE=not estimable; rcPMP=recombinant Escherichia coli -derived cPMP. a Quartile estimates from product-limit (Kaplan-Meier) method, with associated log-log confidence intervals. b Based on the area under the survival curves up to 1 year of follow-up. c Based on the area under the survival curves up to 3 years of follow-up. d Based on Cox proportional hazards model regressing survival status on an indicator variable denoting treatment status.

The 95% CIs are based on the modified score test statistic under the Cox model. The hazard ratio represents the risk of death in the treated patients compared to the untreated historical control patients. NULIBRY (or rcPMP) (n=13) Untreated Genotype-Matched Historical Control (n=18) Treatment Difference (95% CI) Number of Deaths (%) 2 (15%) 12 (67%) 50th Percentile (Median) Survival Time in Months (95% CI) a NE (16, NE) months 48 (10, 99) months Kaplan Meier Survival Probability (95%… [Excerpted — this section continues on DailyMed.]

🧪 Nonclinical Toxicology 188 words ▾

13 NONCLINICAL TOXICOLOGY

13.1Carcinogenesis, Mutagenesis, Impairment of Fertility Carcinogenesis Carcinogenicity studies have not been conducted with fosdenopterin. Mutagenesis Fosdenopterin was not genotoxic in a standard battery of in vitro (bacterial reverse mutation and human lymphocyte chromosomal aberration) and in vivo (rodent bone marrow micronucleus) assays. Impairment of Fertility In a fertility and embryo-fetal development study, subcutaneous administration of fosdenopterin to mice once daily from 14 days prior to mating and continuing through GD 15 at doses up to 200 mg/kg/day (approximately 18 times the MRHD of 0.9 mg/kg/day, based on body surface area) did not adversely affect fertility or reproductive performance.

13.2Animal Toxicology and/or Pharmacology Fosdenopterin has demonstrated phototoxic potential in an animal study at doses equal to and greater than 4.5 times the maximum recommended human dose (based on human equivalent dose comparison). In this study, which was conducted in pigmented rats, intravenous (bolus) administration of fosdenopterin for three consecutive days followed by ultraviolet radiation (UVR) exposure resulted in dose-dependent cutaneous skin reactions (erythema, edema, flaking, and eschar) and ophthalmic and histopathologic changes indicative of phototoxicity [ see Warnings and Precautions ( 5.1 ) ].

📄 Carcinogenesis, Mutagenesis, Impairment of Fertility 99 words ▾

13.1Carcinogenesis, Mutagenesis, Impairment of Fertility Carcinogenesis Carcinogenicity studies have not been conducted with fosdenopterin. Mutagenesis Fosdenopterin was not genotoxic in a standard battery of in vitro (bacterial reverse mutation and human lymphocyte chromosomal aberration) and in vivo (rodent bone marrow micronucleus) assays. Impairment of Fertility In a fertility and embryo-fetal development study, subcutaneous administration of fosdenopterin to mice once daily from 14 days prior to mating and continuing through GD 15 at doses up to 200 mg/kg/day (approximately 18 times the MRHD of 0.9 mg/kg/day, based on body surface area) did not adversely affect fertility or reproductive performance.

📄 Patient Package Insert ~3 min read ▾

INSTRUCTIONS FOR USE NULIBRY ® [noo lye bree] (fosdenopterin) for injection for intravenous use This Instructions for Use contains information on how to prepare and give NULIBRY. Read this Instructions for Use before you mix and give a dose of NULIBRY for the first time and each time you get a NULIBRY refill. There may be new information.

This information does not take the place of talking to your healthcare provider about your child's medical condition or their treatment. Your healthcare provider should show you the right way to mix and give your child's prescribed dose of NULIBRY before you do this for the first time. NULIBRY is given into your child's vein (intravenously), through a special access catheter or port placed by your healthcare provider.

Always follow the specific instructions given by your healthcare provider. If you have questions about preparing or giving NULIBRY, call Sentynl Cares | NULIBRY Patient Support Services at 1-888-251-2800. Important information you need to know before preparing and giving NULIBRY: Your child's dose of NULIBRY is based on their weight.

Your healthcare provider will prescribe the amount of NULIBRY needed for each dose for your child. The amount of NULIBRY needed for each dose and the number of vials needed to prepare each dose may change at each visit with your healthcare provider. The dose will be measured as the amount (volume) of solution needed in milliliters (mL).

Keep a treatment log sheet and write down the: number of vials used to prepare each dose date of each dose of NULIBRY lot number from each NULIBRY vial used total amount (volume) of NULIBRY that was given start time of the dose and the time that the dose was finished Be sure to keep this information up-to-date when the dose changes. Bring your treatment log sheets to each follow-up visit with your healthcare provider. Be sure to have your healthcare provider or pharmacist fill in the following information on your treatment log sheet: your child's dose of NULIBRY in milliliters (mL) number of vials needed to prepare each dose NULIBRY comes as a powder or cake in a vial.

Each vial of NULIBRY must be mixed with Sterile Water for Injection to mix (dissolve) the powder or cake before use. Do not mix NULIBRY with anything other than Sterile Water for Injection . NULIBRY must be given within 4 hours of mixing .

You may keep the mixed solution of NULIBRY at room temperature or refrigerated, for up to 4 hours, until you are ready to give the dose. If you do not give the prepared dose of NULIBRY within 4 hours, all mixed medicine must be thrown away. See “How should I store NULIBRY?” below.

Do not put NULIBRY back in the freezer after it has been mixed. Do not shake NULIBRY after it has been mixed. If your child misses a dose of NULIBRY, give the dose as soon as possible.

Give the next scheduled dose at least 6 hours after you finish giving the missed dose. Avoid exposing NULIBRY to any heat source, such as a microwave or hot water. Do not share needles and syringes.

See the section “ How should I throw away (dispose of) used needles and syringes?” How should I store NULIBRY? Frozen vials : Store NULIBRY in the freezer between −13°F to 14°F (−25°C to −10°C). Keep NULIBRY vials in the original carton to protect from light until you are ready to use it.

Vials of NULIBRY after mixing: Store vials of NULIBRY that have been mixed at room temperature 59°F to 77°F (15°C to 25°C) or in the refrigerator at 36°F to 46 °F (2°C to 8°C), until you are ready to give the dose. Vials of NULIBRY are for 1 time use only. Throw the vial away after use, even if there is medicine left in the vial.

Do not save for later use. Used vials may be thrown away in your household trash. Preparing to give NULIBRY Step 1: Gather Supplies Use a clean, flat work surface.

Remove from the freezer the NULIBRY vials needed to prepare your child's prescribed dose. You may need more than 1 vial to prepare the total amount needed for 1 dose. Allow the NULIBRY vials t… [Excerpted — this section continues on DailyMed.]

📄 Recent Major Changes 13 words ▾

Dosage and Administration ( 2.2 , 2.4 , 2.5 , 2.6 ) 5/2026

📄 Package Label / Principal Display Panel 30 words ▾

Principal Display Panel – 9.5 mg Carton Label NDC 42358-295-01 Nulibry® (fosdenopterin) for injection 9.5 mg/vial For intravenous Use After Reconstitution One Single-Dose Vial Discard unused portion Rx only Figure

Source: FDA Structured Product Labeling, mirrored from DailyMed / openFDA. Prefer the government’s original formatting? View this label on DailyMed ↗

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The pricing shown on our NDC pages comes from CMS NADAC, a voluntary survey of retail community pharmacy invoices. CMS does not publish a NADAC for every NDC — packages outside the retail survey (institutional and hospital products, bulk packages, discontinued items, and many OTC items) may never receive one. A missing price reflects the survey's scope, not this product's actual cost, and does not mean the product is free or unavailable.
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← 42346-0731-85 · Wise 42358-0329-01 · Sentynl →
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